Table 1.
Ongoing AAV Gene Replacement and Gene Supplementation Trials in the Retina
| ClinicalTrials.gov identifier | Disease | Gene | Target cells | Vector | Delivery | Phase | Drug | Start | Estimated Date of completion |
|---|---|---|---|---|---|---|---|---|---|
| NCT03328130 | Autosomal recessive RP | PDE6B | PRs | AAV2/5 | SR | I/II | HORA-PDE6B | 2017 | 2022 |
| NCT03374657 | Autosomal recessive RP | RLBP1 | RPEs | AAV2/8 | SR | I/II | CPK850 | 2018 | 2025 |
| NCT03252847 | X-linked RP | RPGR | PRs | AAV2 | SR | I/II | 2017 | 2020 | |
| NCT03116113 | X-linked RP | RPGR | PRs | AAV2/8 | SR | II/III | 2017 | 2020 | |
| NCT03316560 | X-linked RP | RPGR | PRs | AAV2 | SR | II/III | AGTC-501 | 2018 | 2024 |
| NCT01482195 | RP | MERTK | RPEs | AAV2 | SR | I | 2011 | 2023 | |
| NCT02556736 | Advanced RP | ChR2 | RGCs | AAV2 | IVT | I/IIa | RST-001 | 2015 | 2033 |
| NCT03326336 | Non-syndromic RP | ChrimsonR | RGCs | AAV2(7m8) | IVT | I/IIa | GS030 | 2018 | 2024 |
| NCT03507686 | Choroideremia | REP1 | RPE | AAV2 | SR | II | 2017 | 2020 | |
| NCT03496012 | Choroideremia | REP1 | RPE | AAV2 | SR | III | 2017 | 2020 | |
| NCT02407678 | Choroideremia | REP1 | RPE | AAV2 | SR | II | 2016 | 2021 | |
| NCT02341807 | Choroideremia | REP1 | RPE | AAV2 | SR | I/II | 2015 | 2019 | |
| NCT03001310 | Achromatopsia | CNGB3 | PRs | AAV2/8 | SR | I/II | 2016 | 2019 | |
| NCT03278873 | Achromatopsia | CNGB3 | PRs | AAV | SR | I/II | 2017 | 2023 | |
| NCT02599922 | Achromatopsia | CNGB3 | PRs | AAV2 | SR | I/II | 2016 | 2022 | |
| NCT02935517 | Achromatopsia | CNGA3 | PRs | AAV2 | SR | I/II | AGTC-402 | 2017 | 2021 |
| NCT02610582 | Achromatopsia | CNGA3 | PRs | AAV2/8 | SR | I/II | 2017 | 2021 | |
| NCT00516477 | Leber congential amaurosis | RPE65 | RPEs | AAV2 | SR | I/II | VN | 2007 | 2024 |
| NCT01208389 | Leber congential amaurosis | RPE65 | RPEs | AAV2 | SR | I/II | VN | 2010 | 2026 |
| NCT00999609 | Leber congential amaurosis | RPE65 | RPEs | AAV2 | SR | III | VN | 2012 | 2029 |
| NCT02946879 | Leber congential amaurosis | RPE65 | RPEs | AAV2/5 | SR | I/II | AAV OPTIRPE65 | 2016 | 2023 |
| NCT00481546 | Leber congential amaurosis | RPE65 | RPEs | AAV2 | SR | I | 2007 | 2026 | |
| NCT03597399 | Leber congential amaurosis | RPE65 | RPEs | AAV2 | SR | post-authorization | VN | 2019 | 2029 |
| NCT02416622 | X-linked retinoschisis | RS1 | PRs, BCs | AAV2 | IVT | I/II | 2015 | 2022 | |
| NCT02317887 | X-linked retinoschisis | RS1 | PRs, BCs | AAV2/8 | IVT | I/II | RS1 AAV Vector | 2014 | 2021 |
| NCT02065011 | Usher syndrome 1B | MY07A | PRs | LV | SR | I/II | UshStat | 2013 | 2035 |
| NCT01505062 | Usher syndrome 1B, RP | MY07A | PRs | LV | SR | I/II | SAR421869 | 2012 | 2020 |
| NCT01367444 | Stargardt disease | ABCA4 | PRs | LV | SR | I/II | SAR422459 | 2011 | 2019 |
| NCT01736592 | Stargardt disease | ABCA4 | PRs | LV | SR | I/II | SAR422459 | 2012 | 2034 |
RP, retinitis pigmentosa; AAV, adeno-associated virus; LV, lentivirus; PRs, photoreceptor cells; RGCs, retinal ganglion cells; SR, subretinal injection; RPEs, retinal pigment epithelial cells; IVT, intravitreal injection; VN, voretigene neparvovecrzyl; BCs, bipolar cells.